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Surrogate outcomes in health technology assessment: An international comparison

  • Marcial Velasco Garrido (a1) and Sandra Mangiapane (a2)

Objectives: Our aim was to review the recommendations given by health technology assessment (HTA) institutions in their methodological guidelines concerning the use of surrogate outcomes in their assessments. In a second step, we aimed at quantifying the role surrogate parameters take in assessment reports.

Methods: We analyzed methodological papers and guidelines from HTA agencies with International Network of Agencies for Health Technology Assessment membership as well as from institutions related to pharmaceutical regulation (i.e., reimbursement, pricing). We analyzed the use of surrogate outcomes in a sample of HTA reports randomly drawn from the HTA database. We checked methods, results (including evidence tables), and conclusions sections and extracted the outcomes reported. We report descriptive statistics on the presence of surrogate outcomes in the reports.

Results: We identified thirty-four methodological guidelines, twenty of them addressing the issue of outcome parameter choice and the problematic of surrogate outcomes. Overall HTA agencies call on caution regarding the reliance on surrogate outcomes. None of the agencies has provided a list or catalog of acceptable and validated surrogate outcomes. We extracted the outcome parameter of 140 HTA reports. Only around half of the reports determined the outcomes for the assessment prospectively. Surrogate outcomes had been used in 62 percent of the reports. However, only 3.6 percent were based upon surrogate outcomes exclusively. All of them assessed diagnostic or screening technologies and the surrogate outcomes were predominantly test characteristics.

Conclusions: HTA institutions seem to agree on a cautious approach to the use of surrogate outcomes in technology assessment. Thorough assessment of health technologies should not rely exclusively on surrogate outcomes.

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3.Biomarkers Definition Working Group. Biomarkers and surrogate end points: Preferred definitions and conceptual framework. Clin Pharmacol Ther. 2001;69:8995.

4. HC Bucher , GH Guyatt , DJ Cook , A Holbrook , FA McAlister . Users' guides to the medical literature: XIX. Applying clinical trial results. A. How to use an article measuring the effect of an intervention on surrogate end points. Evidence-Based Medicine Working Group. JAMA. 1999;282:771778.

7.Committee of Principal Investigators. A co-operative trial in the primary prevention of ischaemic heart disease using clofibrate: Report from the Committee of Principal Investigators. Br Heart J. 1978;40:10691118.

9. J Dretzke , C Cummins , J Sandercock , A Fry-Smith , Barrett T, Burls A. Autoantibody testing in children with newly diagnosed type 1 diabetes mellitus. Health Technol Assess. 2004;8:1204.

11. TR Fleming , DL de Mets . Surrogate end points in clinical trials: Are we being misled? Ann Intern Med. 1996;125:605613.

13. DG Fryback , JR Thornbury . The efficacy of diagnostic imaging. Med Decis Making. 1991;11:8894.

22.MERIT-HF Study Group. Effect of metoprolol CR/XL in chronic heart failure: Metoprolol CR/XL randomized intervention trial in congestive heart failure. (MERIT-HF). Lancet. 1999;353:20012007.

29. BM Psaty , NS Weiss , CD Furberg , Surrogate end points, health outcomes and the drug-approval process for the treatment of risk factors for cardiovascular disease. JAMA. 1999;282:786790.

31. J Shepherd , J Jones , D Hartwell , Interferon alfa (pegylated and nonpegylated) and ribavirin for the treatment of mild chronic hepatitis C – a systematic review and economic evaluation. Health Technol Assess. 2007;11:1224.

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International Journal of Technology Assessment in Health Care
  • ISSN: 0266-4623
  • EISSN: 1471-6348
  • URL: /core/journals/international-journal-of-technology-assessment-in-health-care
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